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John Landers, PhD

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John Landers, PhD

University of Massachusetts

Genome-wide Screening of RNAi Towards Developing a Therapeutic for ALS

Over the past several years, there have been great advances in gene therapy to reduce the expression of individual genes as a therapeutic for human disease. Here, we propose to develop a screen of all genes to identify those in which reduced expression is capable of rescuing the diminished survival of neurons expressing mutant ALS genes. Such hits represent therapeutics for ALS as well as providing valuable insight into the pathogenic mechanism of disease.

 

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Steven Finkbeiner

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Gladstone Institutes, UCSF
Two recently discovered genes that have been associated with both familial and sporadic forms of ALS encode the related proteins TDP43 and FUS cause neuron death in ALS.
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